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Treatments for Gaucher Disease

Treatment List for Gaucher Disease

The list of treatments mentioned in various sources for Gaucher Disease includes the following list. Always seek professional medical advice about any treatment or change in treatment plans.

  • Enzyme replacement therapy
  • Enzyme replacement therapy (ERT) for Gaucher disease is now available, with most patients receiving recombinant enzyme (imiglucerase [Cerezyme]). This preparation is highly effective in reversing the visceral and hematologic manifestations of Gaucher disease

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Unlabeled Drugs and Medications to treat Gaucher Disease:

Unlabelled alternative drug treatments for Gaucher Disease include:

Discussion of treatments for Gaucher Disease:

Genes and Disease by the National Center for Biotechnology (Excerpt)

In 1991, enzyme replacement therapy became available as the first effective treatment for Gaucher disease. The treatment consists of a modified form of the glucocerebrosidase enzyme given intravenously. Performed on an outpatient basis, the treatment takes about 1 -- 2 h and is given every 2 weeks. Enzyme replacement therapy can stop and often reverse the symptoms of Gaucher disease, allowing patients to enjoy a better quality of life. (Source: Genes and Disease by the National Center for Biotechnology)

NINDS Gaucher's Disease Information Page: NINDS (Excerpt)

Highly effective enzyme replacement therapy is available for patients with type 1 Gaucher’s disease. This therapy decreases liver and spleen size, reduces skeletal abnormalities, and successfully reverses other manifestations of the disorder including abnormal blood counts. There is currently no effective treatment for severe brain damage that may occur in patients with types 2 and 3. (Source: excerpt from NINDS Gaucher's Disease Information Page: NINDS)

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